Targeting IRAK4 in myelodysplastic syndromes (MDS)
Therapeutic targeting of IRAK4 in MDS
This project aims to block an overactive protein called IRAK4 to help people with myelodysplastic syndromes (MDS), especially those with certain splicing-factor changes.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Albert Einstein College of Medicine NIH-funded |
| Lab location | 1 site (Bronx, United States) |
| Project ID | NIH-11170742 on NIH RePORTER |
What this research studies
This project focuses on a long, overactive form of the IRAK4 protein (called IRAK4-L) that is found in many people with myelodysplastic syndromes (MDS). Researchers will analyze patient bone marrow and blood samples and genetic data to see who makes IRAK4-L. They will test drugs that block IRAK4 in laboratory and animal models and link those results back to patient samples. The aim is to identify which patients could later benefit from IRAK4-targeted treatments.
Who could benefit from this research
Good fit: Ideal candidates would be adults with MDS or MDS/AML who have splicing-factor mutations (like U2AF1 or SF3B1) or whose samples show the IRAK4-L isoform.
Not a fit: People whose disease does not make the IRAK4-L protein or whose leukemia is driven by other mechanisms are unlikely to benefit from IRAK4-targeted approaches.
Why it matters
Potential benefit: If successful, this work could lead to targeted treatments that slow or stop disease progression in MDS patients whose cells express the IRAK4-L protein.
How similar studies have performed: Preclinical studies have shown that inhibiting IRAK4 can block leukemic growth in lab and animal models, while clinical testing in patients is still limited or early-stage.
Where this research is happening
Bronx, United States
- Albert Einstein College of Medicine — Bronx, United States (Active)
Researchers
- Principal investigator: Verma, Amit K. — Albert Einstein College of Medicine
- Study coordinator: Verma, Amit K.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.